PH12020551818A1 - Gene therapy constructs and methods of use - Google Patents
Gene therapy constructs and methods of useInfo
- Publication number
- PH12020551818A1 PH12020551818A1 PH12020551818A PH12020551818A PH12020551818A1 PH 12020551818 A1 PH12020551818 A1 PH 12020551818A1 PH 12020551818 A PH12020551818 A PH 12020551818A PH 12020551818 A PH12020551818 A PH 12020551818A PH 12020551818 A1 PH12020551818 A1 PH 12020551818A1
- Authority
- PH
- Philippines
- Prior art keywords
- methods
- gene therapy
- therapy constructs
- constructs
- improved
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 230000001413 cellular effect Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/38—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against protease inhibitors of peptide structure
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/02—Thioester hydrolases (3.1.2)
- C12Y301/02022—Palmitoyl-protein hydrolase (3.1.2.22)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Toxicology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862664741P | 2018-04-30 | 2018-04-30 | |
US201862688640P | 2018-06-22 | 2018-06-22 | |
US201862744068P | 2018-10-10 | 2018-10-10 | |
PCT/US2019/030076 WO2019213180A1 (en) | 2018-04-30 | 2019-04-30 | Gene therapy constructs and methods of use |
Publications (1)
Publication Number | Publication Date |
---|---|
PH12020551818A1 true PH12020551818A1 (en) | 2021-07-26 |
Family
ID=66669057
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PH12020551818A PH12020551818A1 (en) | 2018-04-30 | 2020-10-30 | Gene therapy constructs and methods of use |
Country Status (16)
Country | Link |
---|---|
US (3) | US10874750B2 (en) |
EP (1) | EP3788064A1 (en) |
JP (2) | JP2021521851A (en) |
KR (1) | KR20210005154A (en) |
CN (1) | CN112513071A (en) |
AU (1) | AU2019261982A1 (en) |
BR (1) | BR112020021962A2 (en) |
CA (1) | CA3098674A1 (en) |
CL (1) | CL2020002809A1 (en) |
CO (1) | CO2020014729A2 (en) |
MX (1) | MX2020011514A (en) |
PH (1) | PH12020551818A1 (en) |
SG (1) | SG11202010533WA (en) |
TW (2) | TWI808169B (en) |
WO (1) | WO2019213180A1 (en) |
ZA (1) | ZA202006905B (en) |
Families Citing this family (14)
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CA3098674A1 (en) * | 2018-04-30 | 2019-11-07 | Amicus Therapeutics, Inc. | Gene therapy constructs and methods of use |
WO2020077114A2 (en) | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
WO2021072372A1 (en) * | 2019-10-10 | 2021-04-15 | Amicus Therapeutics, Inc. | Variant igf2 constructs |
IL293068A (en) * | 2019-11-19 | 2022-07-01 | Asklepios Biopharmaceutical Inc | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
CA3177954A1 (en) * | 2020-05-14 | 2021-11-18 | James M. Wilson | Compositions useful for treatment of pompe disease |
WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
CN117120619A (en) | 2020-07-27 | 2023-11-24 | 沃雅戈治疗公司 | Compositions and methods for treating neurological disorders associated with a deficiency in glucosylceramidase beta |
WO2022104186A1 (en) * | 2020-11-16 | 2022-05-19 | Tavotek Biotherapeutics (Hong Kong) Limited | A shielded and homing bispecific antibody that simultaneously inhibits angiogenic pathway targets and her2 family proteins and uses thereof |
JP2023551911A (en) | 2020-12-01 | 2023-12-13 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Compositions and uses thereof for the treatment of Angelman syndrome |
WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
EP4433490A2 (en) | 2021-11-17 | 2024-09-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
WO2024151982A1 (en) | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
WO2024163012A1 (en) | 2023-02-02 | 2024-08-08 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
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EP1118334A1 (en) | 2000-01-11 | 2001-07-25 | Aventis Behring Gesellschaft mit beschränkter Haftung | Method for the production of conjugates and uses thereof for the prevention and treatment of allergic reactions and autoimmune diseases |
WO2001096584A2 (en) | 2000-06-12 | 2001-12-20 | Akkadix Corporation | Materials and methods for the control of nematodes |
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US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
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2019
- 2019-04-30 CA CA3098674A patent/CA3098674A1/en active Pending
- 2019-04-30 AU AU2019261982A patent/AU2019261982A1/en active Pending
- 2019-04-30 JP JP2020560355A patent/JP2021521851A/en active Pending
- 2019-04-30 TW TW108115137A patent/TWI808169B/en active
- 2019-04-30 WO PCT/US2019/030076 patent/WO2019213180A1/en unknown
- 2019-04-30 MX MX2020011514A patent/MX2020011514A/en unknown
- 2019-04-30 BR BR112020021962-2A patent/BR112020021962A2/en unknown
- 2019-04-30 EP EP19727170.3A patent/EP3788064A1/en active Pending
- 2019-04-30 TW TW112122938A patent/TW202344691A/en unknown
- 2019-04-30 SG SG11202010533WA patent/SG11202010533WA/en unknown
- 2019-04-30 KR KR1020207033891A patent/KR20210005154A/en unknown
- 2019-04-30 CN CN201980029113.5A patent/CN112513071A/en active Pending
- 2019-04-30 US US16/399,979 patent/US10874750B2/en active Active
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2020
- 2020-10-29 CL CL2020002809A patent/CL2020002809A1/en unknown
- 2020-10-30 PH PH12020551818A patent/PH12020551818A1/en unknown
- 2020-11-05 ZA ZA2020/06905A patent/ZA202006905B/en unknown
- 2020-11-27 CO CONC2020/0014729A patent/CO2020014729A2/en unknown
- 2020-12-18 US US17/127,001 patent/US11491243B2/en active Active
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2022
- 2022-11-07 US US18/053,160 patent/US20230233711A1/en active Pending
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2024
- 2024-04-19 JP JP2024068431A patent/JP2024119788A/en active Pending
Also Published As
Publication number | Publication date |
---|---|
EP3788064A1 (en) | 2021-03-10 |
CN112513071A (en) | 2021-03-16 |
US11491243B2 (en) | 2022-11-08 |
CO2020014729A2 (en) | 2020-12-21 |
ZA202006905B (en) | 2022-03-30 |
JP2024119788A (en) | 2024-09-03 |
CL2020002809A1 (en) | 2021-04-30 |
US20190343968A1 (en) | 2019-11-14 |
BR112020021962A2 (en) | 2021-01-26 |
US20230233711A1 (en) | 2023-07-27 |
TW202003053A (en) | 2020-01-16 |
TW202344691A (en) | 2023-11-16 |
SG11202010533WA (en) | 2020-11-27 |
CA3098674A1 (en) | 2019-11-07 |
US10874750B2 (en) | 2020-12-29 |
US20210162075A1 (en) | 2021-06-03 |
AU2019261982A1 (en) | 2020-10-15 |
JP2021521851A (en) | 2021-08-30 |
KR20210005154A (en) | 2021-01-13 |
MX2020011514A (en) | 2020-12-09 |
WO2019213180A1 (en) | 2019-11-07 |
TWI808169B (en) | 2023-07-11 |
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